GeneticsNature CommunicationsPeer-reviewed
Gene Therapy Mediates Therapeutic Improvement in Cardiac Hypertrophy and Survival in a Murine Model of MYBPC3-Associated Cardiomyopathy
Amara Greer-Short, Anna Greenwood, Elena C. Leon…·Laura Marie Lombardi
This study demonstrates that AAV gene therapy with TN-201 restores MYBPC3 protein levels, reverses cardiac hypertrophy, and improves survival in a murine model of MYBPC3-associated HCM.
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